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William Peranteau is the guy parents call when they’ve received the kind of bad news that sinks stomachs and wrenches hearts. Sometimes it’s a shadow on an ultrasound or a few base pairs out of place on a prenatal genetic test, revealing that an unborn child has a life-threatening developmental defect. Pediatric surgeons like Peranteau, who works at Children’s Hospital of Philadelphia, usually can’t try to fix these abnormalities until their patients leave their mother’s bodies behind. And by then it might be too late.
It’s with the memory of the families he couldn’t help in the back of his mind that Peranteau has joined a small group of scientists trying to bring the fast-moving field of gene-editing to the womb. Such editing in humans is a long way off, but a spate of recent advances in mouse studies highlight its potential advantages over other methods of using Crispr to snip away diseases. Parents confronted with an in utero diagnosis are often faced with only two options: terminate the pregnancy or prepare to care for a child that may require multiple invasive surgeries over the course of their lifetime just to survive. Prenatal gene editing may offer a third potential path. “What we see as the future is a minimally invasive way of treating these abnormalities at their genetic origin instead,” says Peranteau.
Vision - Peranteau - Colleagues - University - Pennsylvania
To prove out this vision, Peranteau and colleagues at the University of Pennsylvania injected Crispr editing components, encoded in a virus, into the placentas of pregnant mice whose unborn pups were afflicted with a lethal lung disease-causing mutation. When the fetuses breathed in the amniotic fluid they also inhaled the Crispr bits, which went to work editing the DNA inside their rapidly dividing alveolar progenitor cells. These cells give rise to many types of cells that line the lungs—including...
(Excerpt) Read more at: WIRED
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